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Choosing Outcomes
Across a Population:
Deliberative Approaches
Evan Mayo-Wilson, MPA, DPhil
Center for Clinical Trials and Evidence Synthesis
Department of Epidemiology
Johns Hopkins Bloomberg School of Public Health
[email protected]
Johns Hopkins Bloomberg School of Public Health
Conflict of interest: None
Funding: PCORI, JH-CERSI / FDA, ESRC (UK)
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Core outcomes
“minimum set of outcome measure
that must be reported in all RCTs in a
given health condition”
(Boers, et al., 2014)
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Why core outcomes?
 Enable decisions based on most
important outcomes (& measures)
 Prevent bias
 Facilitate comparison & synthesis
 Reduce waste
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How to identify outcomes?
 Include patient advisors
 Traditional qualitative research (e.g., focus
groups)
 Stated preference methods
 Consensus process (Delphi, nominal group
technique, deliberative methods)
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Patient reported outcomes
 Direct assessment of patient experience
 PROs may be important to patients
 Important outcomes can also be observed
 Some patients cannot report outcomes
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Patient selected outcomes
 Patients may identify what matters
 But not aware of all possible outcomes
(especially new products)
 Medical terms vs. describing experience
Glucose > 120 mg/dl
Increased prolactin levels
Prolongation of the QT interval
Extrapyramidal symptoms
 Values may differ
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Patients make a difference
For example, the original OMERACT core set in
rheumatoid arthritis was established without direct
patient input; but focus groups were held at OMERACT
6 (in 2002), the first OMERACT meeting that patients
were invited to attend. Supported by a previous email
survey, fatigue and sleep were identified as missing
from the OMERACT core set, which only included pain,
function, joint counts, global assessments and a blood
test. The experience of fatigue has been reported by a
large proportion of people with rheumatoid arthritis, and
it is often the most important problem for individual
patients.
(Williamson, et al., 2012)
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Addressing diversity
 Vision not meeting requirement for driving
 Cataracts
Relatively more important to:
1) Whites
2) People with high SES
 Glaucoma
 Needing eye surgery (for cataracts or high eye pressure)
 Needing medicine for controlling high blood pressure or
cholesterol
 Infection (e.g., sinusitis)
Yu T, Holbrook JT, Thorne JE, Flynn TN, Van Natta ML, Puhan MA (2015). ”Outcome preferences in patients with
noninfectious uveitis: Results of a Best-Worst Scaling Study." Invest Ophthalmol Vis Sci 56: 6864-6872.
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www.comet-initiative.org
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Delphi consensus method
Round 1:
Topic generation
Round 2:
Assessment
Round 3:
Feedback / Discussion
Round 4:
Reassessment
 Asynchronous and anonymous
 Large or small groups
 Combines quantitative and
qualitative data
 Patients can propose outcomes
 Iterative
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Importance (n = 20)
Your response
Group
median
Helpfulness (n = 20)
Ease of implementation (n = 20)
Number of panelists
who answered this
question
Hover over to see
the response scale
Timing of Implementation (n = 20)
ExpertLens Software, RAND Corp
Thanks to Dmitry Khodyakov and Sean Grant
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Delphi with multiple groups
Group 1
Group 2
Group 3
Assessment
Assessment
Assessment
Feedback / Discussion
Feedback / Discussion
Feedback / Discussion
Reassessment
Reassessment
Reassessment
Feedback / Discussion
Reassessment
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Conclusions
 Identify important outcomes
 Explore areas consensus &
disagreement
 Compare within and between groups
 Identify hypotheses for confirmatory
research
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